Bone Marrow Transplant

High-dose chemotherapy with bone marrow transplant may still prove to be a viable option for some breast cancer patients and needs to be tested in ongoing clinical trials, according to leading researchers at the National Cancer Institute (NCI) and elsewhere.

In an open letter to physicians -- members of the American Society of Clinical Oncology (ASCO) -- NCI Director Richard Klausner, M.D., urged them not to dismiss bone marrow transplant clinical trials as an option for their breast cancer patients. He said that although preliminary results from some transplant trials have been disappointing, they cannot be generalized to all patients and, contrary to the impression created by some reports, they have not resolved the questions about transplants.

"The largest NCI-sponsored trial is still open but is in dire need of your support," Klausner wrote in the letter, released at ASCO's annual meeting, May 19-23 in New Orleans. This trial, designated S9623, is testing high-dose chemotherapy with transplant in patients with less advanced disease than those in earlier trials. "Importantly, this trial tests the question in a different subset of patients than did prior NCI-sponsored trials, and, accordingly, the preliminary results of these earlier trials may not be applicable to this subset."

Debate about bone marrow transplantation for breast cancer has intensified in the past year, following preliminary reports that the rigorous and costly regimen did not prove superior to standard-dose chemotherapy in several large trials. Enrollment in trials fell in the wake of negative news reports and controversy in the oncology community.

"Emotions regarding transplant continue to run high," Klausner wrote. "The question has been hotly debated in courtrooms, in legislatures, and especially in the media." Most experts believe that the question can be resolved only through data obtained from ongoing clinical trials.

Despite the controversy, most women would be willing to consider the therapy and to enroll in a clinical trial, according to a new survey by NCI and the National Alliance of Breast Cancer Organizations (NABCO). The survey included a nationally representative sample of 925 women ages 35-74.

More than 75 percent of the women who had heard of high-dose chemotherapy with transplant said they would be either very likely (50 percent) or somewhat likely (28 percent) to consider this form of treatment if faced with a decision about breast cancer treatment. The majority of those favorably disposed toward transplants said they would consider enrolling in a clinical trial of high-dose chemotherapy with transplant.

"The survey results show that women have not written off transplant as an option," said Amy S. Langer, Executive Director of NABCO and herself a 14-year breast cancer survivor. NABCO is a leading non-profit U.S. information and education resource on breast cancer.

"However," she added, "in the past, most women with aggressive or advanced breast cancer who turned to bone marrow transplant as their best hope chose the treatment with little information about its efficacy. Only by completing the remaining transplant trials will we be able to offer sound guidance about which treatments work best and permit tomorrow's patients to make choices based on fact, rather than emotion."

Other leaders in oncology have also urged continued study of this treatment. In an editorial published several days ago in Cancer Investigation, Scott Bearman, M.D., warned against sweeping conclusions that HDC-ABMT does not work. "Many more trials will be required before a consensus about the value of dose intensity for breast cancer emerges," said Bearman, a leading oncologist at the University of Colorado Health Sciences Center, Denver, and principal investigator of S9623.

Many patient advocacy organizations have taken similar positions. "It would be shortsighted to close the door on all transplant trials for breast cancer based on the information we have right now," said Susan Braun, president and chief executive officer of The Susan G. Komen Breast Cancer Foundation.

NCI's current trial, S9623, continues to be offered to breast cancer patients who have four or more lymph nodes positive for cancer and who are at high risk of recurrence. Women are randomized to one of two high-dose regimens -- high-dose chemotherapy with transplant or high-dose chemotherapy with granulocyte colony stimulating factor (G-CSF) to stimulate white blood cell production.

"This is the largest U.S. trial looking at transplant in women with less advanced disease," said Jeff Abrams, M.D., who coordinates breast cancer trials for the NCI's Cancer Therapy Evaluation Program. "The hypothesis that a transplant may benefit certain groups of patients is still very much an open question. One of our best shots at improved treatment for certain stages of breast cancer is to complete this trial."

A copy of Dr. Klausner's letter and more information on S9623 and the NCI/NABCO survey are available are available through NCI's Web site for clinical trials, http://cancertrials.nci.nih.gov, or call the NCI press office at 301-496-6641.